TUESDAY, AUGUST 25, 2009
The Glimmer of Hope
I posted a link about the bone marrow transplants that are being done in the states for kids with Recessive Dystrophic EB (Kourtney's type of EB). I really feel that I need to update you on where we stand in the process of all of this.
2 years ago we were sent information about the Liao family. They had 2 boys with RDEB and in a mom's desperate attempt to make her boys well, she went out on a limb and convinced a hematologist to try a stem cell transplant on her eldest son with RDEB. We followed this story with interest and in Spring 2008 there was an article in the Vancouver Sun that suggested that this boy had been cured of the RDEB. As we did more research and talked to more doctors we were very encouraged that the bone marrow transplant was successful. There was a clinical trial that was being put into place and they were looking for more participants. This of course sparked our interest and we began emailing the nurse at a hospital in Minnesota. We found out that the transplant alone would cost 1 million dollars but if there was a sibling bone marrow match the cost would be 500,000 dollars. So last summer we all got tested at Children's Hospital. We found out that Kourtney and Shaelyn were HLA (bone marrow) matches to each other. We were so amazed by all this and really felt that the door was being opened up for us to go to Minneapolois to get this done (as most of you know Shaelyn was diagnosed with RDEB through amniocentesis which carried a 100 percent accuracy rate, and we were highly encouraged to abort.) We did not think that this was a coincidence and even felt more led to pursue the clinical trial.
A couple of months later we began to hear criticism about this trial as one child died during the preparation for the bone marrow transplant. The other criticism we heard was that Mrs. Liao was just saying her son was doing better, but clinically he wasn't doing better according to the "twitter for doctors". I could understand the skepticism but also felt that she must have been encouraged enough to put her 2nd child with RDEB through the same procedure. It was very difficult for me to evaluate all this information but I felt very strongly that I needed to pursue this. I had all my papers ready to send to Minneapolis and then Kourtney got sick last August.
Over the last year we have gotten to know a hematologist at Children's Hospital (which to me has been a divine turn of events). He has close contact with what is going on in Minneapolis but has cautioned that bone marrow transplants are very nasty and according to him, the clinical benefits for kids with EB do not outweigh the risks associated with a bone marrow transplant. Yet he has not seen Kourtney without her bandages so I am not sure if he really understands how nasty EB is.
Over the last year I have emailed the nurse in Minneapolis and did not hear a reply so I was under the impression that the clinical trial had been discontinued. This last month there has been a update on CNN on the Liao family and also there have been two other children featured in newspapers who have had the bone marrow transplant done with successful results.
Although I would love to take Kourtney down to get this done I also realize that there are huge implications for doing this. At this point Kourtney is not strong emotionally and physically to have this done. I also know that I, myself, have "few" reserves left after our year spent in the hospital and don't feel emotionally ready to be in a city in the states without any support around. Kourtney and I experienced loneliness being at Children's Hospital and could not imagine being in a city where we didn't know anyone. The money thing is a huge drawback, although it could be done with fundraisers, we could sell our house and downsize, but we still have to keep in mind that this procedure has been proven not to be a cure for EB, but it does help with the symptoms of EB. We would still have to have a special bathtub and a dressing room, I couldn't imagine starting from nothing again especially when our home is so designed for Kourtney's special needs.
We just need continued prayer and support for our family. Although Kourtney is doing soooo much better we are still facing the tween years and many more battles to come. We are hoping we have seen the worse and could not imagine it getting any more difficult.
2 years ago we were sent information about the Liao family. They had 2 boys with RDEB and in a mom's desperate attempt to make her boys well, she went out on a limb and convinced a hematologist to try a stem cell transplant on her eldest son with RDEB. We followed this story with interest and in Spring 2008 there was an article in the Vancouver Sun that suggested that this boy had been cured of the RDEB. As we did more research and talked to more doctors we were very encouraged that the bone marrow transplant was successful. There was a clinical trial that was being put into place and they were looking for more participants. This of course sparked our interest and we began emailing the nurse at a hospital in Minnesota. We found out that the transplant alone would cost 1 million dollars but if there was a sibling bone marrow match the cost would be 500,000 dollars. So last summer we all got tested at Children's Hospital. We found out that Kourtney and Shaelyn were HLA (bone marrow) matches to each other. We were so amazed by all this and really felt that the door was being opened up for us to go to Minneapolois to get this done (as most of you know Shaelyn was diagnosed with RDEB through amniocentesis which carried a 100 percent accuracy rate, and we were highly encouraged to abort.) We did not think that this was a coincidence and even felt more led to pursue the clinical trial.
A couple of months later we began to hear criticism about this trial as one child died during the preparation for the bone marrow transplant. The other criticism we heard was that Mrs. Liao was just saying her son was doing better, but clinically he wasn't doing better according to the "twitter for doctors". I could understand the skepticism but also felt that she must have been encouraged enough to put her 2nd child with RDEB through the same procedure. It was very difficult for me to evaluate all this information but I felt very strongly that I needed to pursue this. I had all my papers ready to send to Minneapolis and then Kourtney got sick last August.
Over the last year we have gotten to know a hematologist at Children's Hospital (which to me has been a divine turn of events). He has close contact with what is going on in Minneapolis but has cautioned that bone marrow transplants are very nasty and according to him, the clinical benefits for kids with EB do not outweigh the risks associated with a bone marrow transplant. Yet he has not seen Kourtney without her bandages so I am not sure if he really understands how nasty EB is.
Over the last year I have emailed the nurse in Minneapolis and did not hear a reply so I was under the impression that the clinical trial had been discontinued. This last month there has been a update on CNN on the Liao family and also there have been two other children featured in newspapers who have had the bone marrow transplant done with successful results.
Although I would love to take Kourtney down to get this done I also realize that there are huge implications for doing this. At this point Kourtney is not strong emotionally and physically to have this done. I also know that I, myself, have "few" reserves left after our year spent in the hospital and don't feel emotionally ready to be in a city in the states without any support around. Kourtney and I experienced loneliness being at Children's Hospital and could not imagine being in a city where we didn't know anyone. The money thing is a huge drawback, although it could be done with fundraisers, we could sell our house and downsize, but we still have to keep in mind that this procedure has been proven not to be a cure for EB, but it does help with the symptoms of EB. We would still have to have a special bathtub and a dressing room, I couldn't imagine starting from nothing again especially when our home is so designed for Kourtney's special needs.
In all honesty I believe it will happen one day but it will happen at Children's Hospital and it will be covered by our health care. I believe there will be a clinical reason to get it done and that reason is her anemia issues that I blogged about a month ago. A bone marrow transplant would replace her tired worn bone marrow with healthy under worked bone marrow.
Right now....we will just take it one day at a time. I know that when the time is right it will be made very clear to us. We want to have HOPE but we want to have "clear" direction when we need to pursue this. I hope this clarifies this for anyone that is wondering and for those eager to fund raise. We are holding back at the moment until we know the time is right. (We would know the time was right if 500,000 fell out of the sky, but at present that has not happened).
We just need continued prayer and support for our family. Although Kourtney is doing soooo much better we are still facing the tween years and many more battles to come. We are hoping we have seen the worse and could not imagine it getting any more difficult.
